A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease
This study is investigating a medicine called Lucerastat for adults living with Fabry disease. The main goal is to understand its long-term safety and how well people tolerate it. Researchers are also looking closely at how Lucerastat affects the kidneys, specifically checking for certain sugar build-up (Gb3 inclusions) in men who have been on the treatment for at least two years. This is a Phase 3 study, meaning it's a later stage of research that helps scientists understand if new treatments are safe and effective. The study aims to gather important information that will help doctors and patients make informed decisions about managing Fabry disease in the future.
At a glance
What is this study about?
This research study is looking into a new medication called Lucerastat for adults who have Fabry disease. Fabry disease is a rare condition that can affect many parts of the body, including the kidneys, heart, and skin. The study's main purpose is to find out if Lucerastat is safe to take over a long period and how well people tolerate it, meaning if they can take it without too many side effects.
Researchers are particularly interested in how Lucerastat affects the kidneys. They will be examining kidney samples from some male participants to see if the medicine helps reduce certain harmful deposits (called Gb3 inclusions) that can build up in the kidneys due to Fabry disease. This detailed look at kidney health will help scientists understand if Lucerastat can make a real difference in preventing some of the serious problems associated with the disease.
This study is designed to continue until Lucerastat becomes widely available or until participants leave the study for other reasons. For those in Europe, participation could last for up to eight years, giving researchers a very long-term view of the medicine's effects. The information gathered from this study will be crucial in deciding if Lucerastat could be a new and important treatment option for people with Fabry disease.
Key takeaways
- This study is testing the long-term safety and tolerability of Lucerastat for adults with Fabry disease.
- Researchers are carefully monitoring how the medication affects kidney health over time.
- Participation could last up to 8 years for those in Europe.
- It's a continuation study, so participants must have completed an earlier related study.
- The findings will help determine if Lucerastat is a safe and effective long-term treatment option.
Who may be eligible?
To be considered for this study, you must be an adult aged 18 or older with Fabry disease and have already completed the first six months of treatment in an earlier related study (ID 069A301). You'll need to sign a consent form, showing you understand the study and agree to take part.
If you are a woman who could become pregnant, you'll need to agree to use effective birth control and take monthly pregnancy tests. If you are a man who could father a child, you'll need to agree to use a condom and not try to have children while on the study. Anyone who is pregnant, planning to become pregnant, or breastfeeding cannot join.
There are also some health conditions that would prevent you from joining. For example, if your doctor thinks you are at high risk of developing serious organ problems soon, or if you have any other illness that might make it hard to follow the study rules or understand the results. Also, if you experienced certain serious kidney, stroke, or heart problems during the previous study, you would not be able to join this long-term study.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you 18 years old or older?
- Do you have Fabry disease?
- Have you completed the previous 6-month study (ID 069A301)?
- Are you a woman who agrees to use birth control and take monthly pregnancy tests if you could become pregnant?
- Are you a man who agrees to use a condom and not father a child if you could?
- Are you not pregnant, planning pregnancy, or breastfeeding?
What does participation involve?
Taking part in this study means you'll continue to take the study medicine, Lucerastat, as prescribed. You'll have regular visits to the clinic for check-ups, which will include blood tests, urine tests, and other assessments to monitor your health and how the medicine is affecting you. For some male participants with classic Fabry disease, there will also be a sub-study involving kidney tissue checks after at least two years of treatment with Lucerastat. The total time you might be in the study can vary, for those in Europe, it could be up to 8 years (96 months). You'll have ongoing contact with the study team to ensure your safety and that the study is running smoothly. The exact number and timing of visits will be explained in detail by the study staff.
Potential risks and benefits
Locations (42)
- University of Alabama at Birmingham - Nephrology Research ClinicVerified postcodeBirmingham, United States
- University of California IrvineVerified postcodeIrvine, United States
- UCSF Benioff Children's Hospital OaklandVerified postcodeOakland, United States
- University of Florida Clinical and Translational Science Institute, UF Clinical Research CenterVerified postcodeGainesville, United States
- Rush University Medical Center - Dept of PediatricsVerified postcodeChicago, United States
- University of Iowa Stead Family Children's Hospital - Division of Medical GeneticsVerified postcodeIowa City, United States
- Massachusetts General HospitalVerified postcodeBoston, United States
- Infusion AssociatesVerified postcodeGrand Rapids, United States
- University of Pennsylvania - Dept of MedicineVerified postcodePhiladelphia, United States
- Greenwood Genetics CenterVerified postcodeGreenville, United States
- Renal Disease Research Institute LLCVerified postcodeDallas, United States
- Baylore University Medical CenterVerified postcodeDallas, United States
Common questions
What is Lucerastat?
Lucerastat is a new medicine being studied for adults with Fabry disease. This study is looking at its long-term safety and whether it helps with the condition.
What is Fabry disease?
Fabry disease is a rare genetic condition that can cause problems in different parts of the body, including the kidneys and heart, due to a build-up of certain fatty substances.
How long will I be in the study?
For participants in Europe, the study could last up to 8 years. The exact duration will be discussed with you by the study team.
Will I have to have any special tests?
Yes, you'll have regular blood and urine tests. Some male participants will also have kidney tissue checks as part of a detailed sub-study.
Can I leave the study at any time?
Yes, you can choose to leave the study at any point, and your decision will not affect your regular medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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